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公开(公告)号:US20230203484A1
公开(公告)日:2023-06-29
申请号:US17926967
申请日:2021-05-24
Applicant: WAVE LIFE SCIENCES LTD.
Inventor: Chandra VARGEESE , Naoki IWAMOTO , Luciano H. APPONI , David Charles Donnell BUTLER , Pachamuthu KANDASAMY , Subramanian MARAPPAN , Snehlata TRIPATHI , Wei LIU , Mugdha BEDEKAR , Vinod VATHIPADIEKAL
IPC: C12N15/113
CPC classification number: C12N15/113 , C12N2310/14 , C12N2310/315 , C12N2310/351
Abstract: The present disclosure provides double stranded oligonucleotides, compositions, and methods relating thereto. The present disclosure encompasses the recognition that structural elements of double stranded oligonucleotides, such as base sequence, chemical modifications (e.g, modifications of sugar, base, and/or internucleotidic linkages) or patterns thereof, and/or stereochemistry (e.g., stereochemistry of backbone chiral centers (chiral internucleotidic linkages)), and/or patterns thereof, can have significant impact on oligonucleotide properties and activities, e.g, RNA interference (RNAi) activity, stability, delivery, etc. The present disclosure also provides methods for treatment of diseases using provided double stranded oligonucleotide compositions, for example, in RNA interference.